Ocular Therapeutix(OCUL) - 2025 Q2 - Quarterly Results

Executive Summary & Business Highlights Company Overview and Strategic Vision Ocular Therapeutix is entering a pivotal phase, focusing on redefining retina treatment with AXPAXLI™, which aims for a powerful and differentiated clinical profile, potentially securing an unprecedented superiority label in wet AMD with long-term dosing flexibility. The company maintains a strong financial position with a cash runway into 2028 - AXPAXLI™ is expected to achieve a powerful and highly differentiated clinical profile, with the potential to secure an unprecedented superiority label in wet AMD[2] - The SOL program is anticipated to enable AXPAXLI™ dosing every 6 months to as infrequently as every 12 months[2] Cash Balance as of June 30, 2025 | Metric | Amount (Millions) | | :----- | :---------------- | | Cash Balance | $391 | - Expected cash runway extends into 2028, well beyond anticipated topline data for SOL-1 and SOL-R[1] Recent Achievements and Upcoming Milestones Ocular Therapeutix reported significant progress in its AXPAXLI™ clinical trials for wet AMD, including SOL-1 remaining on track for 1Q 2026 topline data and streamlined rescue criteria for SOL-R, with data expected in 1H 2027. The company also plans a long-term extension study, received positive FDA feedback for NPDR and DME programs, raised $97 million, and will host an Investor Day in September 2025 AXPAXLI™ Clinical Development - SOL-1 (Phase 3, wet AMD) topline data remains on track for 1Q 2026, with exceptional patient retention and protocol adherence[3] - SOL-R (Phase 3, wet AMD) rescue criteria were streamlined and simplified to align with real-world clinical decision-making (criteria: >5-letter loss in visual acuity plus ≥75-micron increase in central subfield thickness). Topline data is expected in 1H 2027[3] - New Drug Application (NDA) filing for AXPAXLI™ is planned shortly after SOL-R topline results, utilizing the 505(b)(2) pathway for potentially shorter review[3] - A single long-term, open-label extension study is planned for patients completing either SOL-1 or SOL-R to provide long-term safety data and inform the AXPAXLI™ treatment paradigm[4] Pipeline Expansion (NPDR, DME) - Received written feedback from FDA supporting a registrational trial for AXPAXLI™ in non-proliferative diabetic retinopathy (NPDR)[4] - Actively defining clinical strategy for AXPAXLI™ in NPDR and diabetic macular edema (DME), with more details to be shared at Investor Day[4] Corporate and Financial Updates - Raised gross proceeds of approximately $97 million in June 2025 through an existing at-the-market (ATM) facility, extending financial flexibility and cash runway into 2028[4] - Ocular will host an Investor Day on Tuesday, September 30, 2025, in New York City, to discuss SOL trials, the extension study, NPDR/DME strategy, and the global commercial opportunity for AXPAXLI™[4] - Unveiled new corporate branding in June 2025, reflecting the company's transformation to a retina-focused entity committed to redefining the retina experience[4] Financial Performance (Q2 2025) Key Financial Highlights As of June 30, 2025, Ocular Therapeutix reported total cash and cash equivalents of $391.1 million, which is projected to fund operations into 2028, well beyond the anticipated topline data for its key clinical trials Cash and Cash Equivalents (as of June 30, 2025) | Metric | Amount (Millions) | | :----- | :---------------- | | Cash and cash equivalents | $391.1 | - Current cash balance is sufficient to support planned expenses, debt service, and capital expenditure requirements into 2028[5] Revenue Analysis Total net revenue for Q2 2025 decreased by 18.1% year-over-year to $13.5 million, primarily due to a challenging reimbursement environment for DEXTENZA. Despite this, DEXTENZA end-user unit sales increased by 5% YoY, and net product revenue grew by 26.0% quarter-over-quarter Total Net Revenue (Q2 2025 vs Q2 2024) | Metric | Q2 2025 (Millions) | Q2 2024 (Millions) | YoY Change | | :----- | :----------------- | :----------------- | :--------- | | Total Net Revenue | $13.5 | $16.4 | -18.1% | - DEXTENZA end-user unit sales increased by 5% compared to Q2 2024[7] - DEXTENZA net product revenue increased by 26.0% compared to Q1 2025[7] - The reduction in net revenue was attributed to an evolving and significantly more challenging reimbursement environment for DEXTENZA in 2025[7] Operating Expenses Research and development expenses significantly increased by 76.8% YoY to $51.1 million in Q2 2025, driven by increased clinical activities for the SOL-1 and SOL-R trials. Selling and marketing expenses also rose by 37.0% to $13.7 million due to pre-commercialization efforts for AXPAXLI™. General and administrative expenses decreased by 27.4% to $14.3 million, primarily due to one-time costs in the prior year Operating Expenses (Q2 2025 vs Q2 2024) | Expense Category | Q2 2025 (Millions) | Q2 2024 (Millions) | YoY Change | | :--------------- | :----------------- | :----------------- | :--------- | | R&D Expenses | $51.1 | $28.9 | +76.8% | | S&M Expenses | $13.7 | $10.0 | +37.0% | | G&A Expenses | $14.3 | $19.7 | -27.4% | - Increase in R&D expenses reflects higher overall clinical expenses associated with the SOL-1 and SOL-R Phase 3 clinical trials, as well as additional personnel and professional services[8] - Increase in S&M expenses primarily reflects higher personnel-related costs and professional fees for AXPAXLI™ pre-commercialization activities[9] - Decrease in G&A expenses was primarily due to lower personnel-related costs, as Q2 2024 included one-time costs for departed employees and restructuring[10] Net Loss and EPS Ocular Therapeutix reported a net loss of $(67.8) million, or $(0.39) per share, for Q2 2025, an increase from $(43.8) million, or $(0.26) per share, in the comparable quarter of 2024, primarily driven by increased operating expenses Net Loss and EPS (Q2 2025 vs Q2 2024) | Metric | Q2 2025 (Millions) | Q2 2024 (Millions) | YoY Change | | :----- | :----------------- | :----------------- | :--------- | | Net Loss | $(67.8) | $(43.8) | +54.8% | | Net Loss per Share (Basic & Diluted) | $(0.39) | $(0.26) | +50.0% | - Outstanding shares as of August 1, 2025, were approximately 174.0 million[12] Product and Disease Background About AXPAXLI™ AXPAXLI™ (OTX-TKI) is an investigational, bioresorbable, intravitreal hydrogel that incorporates axitinib, a multi-target tyrosine kinase inhibitor with anti-angiogenic properties. It is currently being evaluated for the treatment of wet AMD, diabetic retinopathy, diabetic macular edema, and other retinal diseases - AXPAXLI™ (OTX-TKI) is an investigational, bioresorbable, intravitreal hydrogel[13] - It incorporates axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties[13] - AXPAXLI™ is being evaluated for wet AMD, diabetic retinopathy, diabetic macular edema, and other retinal diseases[13] About SOL-1 Study The SOL-1 trial is a registrational Phase 3 superiority study evaluating AXPAXLI™ against aflibercept (2 mg) in 344 treatment-naïve wet AMD subjects. The primary endpoint is the proportion of subjects maintaining visual acuity at Week 36, with durability evaluated up to Week 52. The study is conducted under an FDA Special Protocol Assessment (SPA) - SOL-1 is a registrational Phase 3, multi-center, double-masked, randomized (1:1), parallel group superiority study[14][15] - The trial completed randomization of 344 evaluable treatment-naïve subjects with wet AMD[14] - The primary endpoint is the proportion of subjects who maintain visual acuity (loss of <15 ETDRS letters of BCVA) at Week 36[16] - The study is being conducted under a Special Protocol Assessment (SPA) agreement with the FDA[16] About SOL-R Study The SOL-R trial is a registrational Phase 3 non-inferiority study designed to evaluate AXPAXLI™ in approximately 555 wet AMD subjects. The primary endpoint is to demonstrate non-inferiority in mean BCVA change from baseline between the AXPAXLI™ and on-label aflibercept (2 mg) arms at Week 56, with a pre-specified non-inferiority margin of -4.5 letters - SOL-R is a registrational Phase 3, multi-center, double-masked, randomized (2:2:1), three-arm non-inferiority study[17][19] - The trial intends to randomize approximately 555 subjects who are treatment-naïve or recently diagnosed with wet AMD[17] - The primary endpoint is to demonstrate non-inferiority in mean BCVA change from baseline between the AXPAXLI™ and on-label aflibercept (2 mg) arms at Week 56[19] - The non-inferiority margin for the lower bound is -4.5 letters of mean BCVA, as agreed with the FDA[19] - Pre-specified rescue criteria include a >5-letter loss in visual acuity plus a ≥75-micron increase in central subfield thickness (CSFT)[18] About Wet AMD Wet age-related macular degeneration (wet AMD) is a leading cause of severe, irreversible vision loss, affecting millions globally. It is characterized by abnormal blood vessel growth and hyperpermeability, primarily stimulated by VEGF. Current treatments involve frequent intraocular injections, which often lead to patient discontinuation and undertreatment, highlighting the need for more sustainable solutions - Wet AMD affects approximately 14.5 million individuals globally and 1.8 million in the United States[20] - It causes vision loss due to abnormal new blood vessel growth and hyperpermeability in the macula, primarily stimulated by vascular endothelial growth factor (VEGF)[20] - Challenges with current therapies include pulsatile, repeated intraocular injections, treatment-related adverse events, and up to 40% patient discontinuation within one year, leading to undertreatment and lack of long-term vision improvement[20] Corporate Information About Ocular Therapeutix, Inc. Ocular Therapeutix is an integrated biopharmaceutical company focused on redefining the retina experience through its proprietary ELUTYX™ bioresorbable hydrogel technology. Its pipeline includes AXPAXLI™ (Phase 3 for wet AMD), the commercial product DEXTENZA (for ocular inflammation/pain/itching), and OTX-TIC (Phase 2 for open-angle glaucoma/ocular hypertension) - Ocular Therapeutix is an integrated biopharmaceutical company committed to redefining the retina experience[21] - The company leverages its ELUTYX™ proprietary bioresorbable hydrogel-based formulation technology[21] - Key products and candidates include AXPAXLI™ (Phase 3 for wet AMD), DEXTENZA (FDA-approved for ocular inflammation, pain, and allergic conjunctivitis), and OTX-TIC (Phase 2 for open-angle glaucoma or ocular hypertension)[21][22] Forward-Looking Statements This section outlines that statements regarding future expectations, plans, and prospects are forward-looking and subject to substantial risks and uncertainties. Actual results may differ materially from those indicated, and the company disclaims any obligation to update these statements unless required by law - Statements about future expectations, plans, and prospects constitute forward-looking statements[24] - Actual results may differ materially from forward-looking statements due to various important factors, risks, and uncertainties[24] - The company disclaims any obligation to update these forward-looking statements, except as required by law[24] Investor Relations This section provides contact information for investor and media inquiries - Contact for Investors & Media: Bill Slattery, Vice President, Investor Relations[25] Financial Statements Consolidated Balance Sheets The Consolidated Balance Sheets present the company's financial position as of June 30, 2025, and December 31, 2024, detailing assets, liabilities, and stockholders' equity. Total assets slightly decreased from $457.9 million to $451.3 million, while total liabilities increased from $142.6 million to $145.4 million, and total stockholders' equity decreased from $315.3 million to $305.9 million Consolidated Balance Sheets (Selected Data in thousands) | Metric | June 30, 2025 | December 31, 2024 | | :-------------------- | :------------ | :---------------- | | Assets | | | | Cash and cash equivalents | $391,134 | $392,102 | | Total current assets | $433,375 | $440,987 | | Total assets | $451,333 | $457,935 | | Liabilities | | | | Total current liabilities | $42,931 | $41,354 | | Total liabilities | $145,417 | $142,591 | | Stockholders' Equity | | | | Total stockholders' equity | $305,916 | $315,344 | Consolidated Statements of Operations and Comprehensive Loss The Consolidated Statements of Operations and Comprehensive Loss detail the company's financial performance for the three and six months ended June 30, 2025, and 2024. For Q2 2025, total net revenue was $13.5 million, operating expenses were $81.1 million, resulting in a net loss of $(67.8) million, or $(0.39) per share. This represents an increased net loss compared to Q2 2024 Consolidated Statements of Operations (Selected Data in thousands, except per share) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Total revenue, net | $13,459 | $16,441 | $24,156 | $31,215 | | Total costs and operating expenses | $81,100 | $60,031 | $155,715 | $106,422 | | Loss from operations | $(67,641) | $(43,590) | $(131,559) | $(75,207) | | Net loss | $(67,814) | $(43,777) | $(131,867) | $(108,625) | | Net loss per share, basic | $(0.39) | $(0.26) | $(0.77) | $(0.73) | | Weighted average common shares outstanding, basic | 172,594,662 | 165,824,778 | 171,004,629 | 148,922,937 |